UAE girl needs worlds most expensive life-saving drug

SMA affects the motor neurons, or the nerve cells in the spinal cord that controls muscle movement. A viral infection like common cold could lead to breathing problems and an abnormally high heart rate that would immediately land Arya in the intensive care unit.

Even at such a young age, she has already made several visits to the ICUs and emergency rooms and knows by heart all her health issues.

“I have been fighting heart attacks, kyphosis and scoliosis,” said the girl, who suffers from 40 degrees of kyphosis, an increased front-to-back curve of the upper spine, and seven degrees of scoliosis, an abnormal curve of the spine.

Arya was diagnosed with the inherited disease when she was one. And ever since, life hasn’t been easy for her, as the damage to her organs has been getting worse.

She can be cured with Zolgensma — but it’s the most expensive life-saving drug in the world, costing Dh8 million. And this hefty price tag has been pushing Arya to become a scientist one day.

“Why is it the costliest? My parents cannot afford it. I want to make a cheaper drug. I will also make a robot for kids with SMA. So, they will be able to run and play like other kids,” the kid said while sitting in her customised wheelchair.

Arya’s SMA battle has been an unforgettable one for her Indian expat parents Astha Muthoo and Rahul Pathak, who were found to be SMA carriers but without any symptoms.

“She stopped crawling when she was one. In early 2016, type 2 SMA was confirmed but there was no medicine available. We were devastated,” Astha said.

Since 2017, Arya has fought many health complications and spent weeks at the hospital.

“A simple cold would turn into pneumonia. She has issues with swallowing. Food can go into her lungs and cause infection. Her lung capacity is less. While lying down, instead of breathing in, she breathes out. Once she started going to school, there were multiple visits to ICUs, and she was on bipap support. With several visits, at times, doctors struggle to find veins on her hands to give drips,” the mother said, citing a bunch of medical reports.

Astha, who left her teaching job, does physiotherapy sessions to improve Arya’s movement.

“She can’t lift her hands or move her muscles. She can’t stand up even with our support.”

Currently, there are three drugs available: Zolgensma, a kind of gene therapy and a one-time dose, and the other two Spinraza (Dh3.4 million) and Risdiplam (Dh1.7 million) have to be taken multiple times for the rest of her life.

“Zolgensma is our best option, but we can’t afford it. We started a fundraiser in India: www.impactguru.com/fundraiser/help-arya-pathak,” Pathak, a private sector employee, said about the initiative, which has hit four per cent funds in the last 23 days.

A paediatric neurorehabilitation consultant at Al Jalila Children’s Specialty Hospital, in his medical report, said: “I prefer the gene therapy, followed by Spinraza. Risdiplam is the preferred last option. As she has had many chest infections before, there is always the risk that with time, she may become weaker and develop respiratory compromise and become oxygen dependent or lose her ability to feed and swallow. The treatment is a lifesaving intervention before she starts to develop any respiratory compromise or feeding problems.”

According to experts, most children with type 2 SMA survive into adulthood.

All such uncertainties and challenges haven’t stopped Arya from going to school in a wheelchair.

“I am a Grade 1 student at Abu Dhabi’s Emirates Private School. I like to read books. One day, I will walk and play like others,” Arya said, showing a beautiful painting of her in a wheelchair and standing on her feet after getting the drug.

Recent news of kids receiving the drug through successful crowdfunding initiatives gives the family hope, as they race against time to give Arya her dose of Zolgensma.

Astha said: “She keeps watching success stories of other kids who have got Zolgensma. This is a lifesaver. It is approved for kids weighing less than 21kg. She is more than 19 kg. It will be a great blessing if we get this drug. She will stay alive.”

ashwani@khaleejtimes.com